Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.
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The two trials evaluating lacutamab, an investigational therapy developed to treat T-cell lymphomas, were put on partial hold by the FDA. The patient died of hemophagocytic lymphohistiocytosis, which is…
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Two CTCL Trails on Partial Hold After Patient Death
One of the most successful healthcare services benefiting the public is newborn screening. Unfortunately, this life-saving health initiative is not provided for all rare diseases in some U.S. states. As…
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Newborn Screening Saves Lives and Lowers Costs
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Genomics is revolutionizing healthcare processes, offering the potential to enhance the lives of numerous individuals by enabling the early detection of treatable disorders and providing lifesaving therapies. Every year, thousands…
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Over 200 Rare Diseases to be Included in Genomics England Research Study
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After years of seeking approval for the treatment of Pompe disease, this week, as reported by GlobeNewswire, the FDA granted approval to its developer, Amicus Therapeutics, for the first…
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FDA Grants Approval for Innovative Dual-Compound Therapy in Pompe Disease
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This week, the USFDA granted a new marketing authorization for the ICHC Cancers Panel, thereby creating a new regulatory classification. According to a report in Inside Precision Medicine, the…
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The FDA Has Approved the First Blood Test to Identify Multiple Hereditary Cancers
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Interferon beta was the first available therapy for treating multiple sclerosis (MS) and is still the most commonly prescribed treatment. This therapy helps reduce relapses in MS patients. Distinguished UCLA…
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Heligenics Finds Variants of IFNB1, Used to Treat Multiple Sclerosis
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For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…
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Death of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered Gene Therapy
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Early in the 1990s, anti-CD20 antibody therapy marked a significant improvement in the outcomes for patients with B-cell cancers. B-cell lymphoma is a type of cancer that originates in…
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Hairy Cell Leukemia: Drug Combo Achieves Significant Remissions
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Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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Cervical Cancer is on the Decline Worldwide but on the Rise in India
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This week Ionis Pharmaceuticals, Inc. issued a press release via PRNewswire announcing topline Phase 3 results for its Balance study of olezarsen. The study ( NCT04568434 ) enrolled people with…
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Ionis Pharmaceuticals Announces Positive Results in Familial Chylomicronemia Syndrome
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In healthy individuals, copper is absorbed from food, and the excess is excreted by a substance called bile which is produced in the liver. It should be noted that…
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Wilsons Disease: Researchers Survey Care of Patients in German University Centers
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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Osaka University Researchers Study Reasons for Rare Disease Diagnostic Delays
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As reported in MedCity News, Ojjaara was part of GSK’s $1.9 billion acquisition of Sierra Oncology last year. FDA approval of GSK’s momelotinib covers the treatment of adult myelofibrosis…
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Newly Approved Treatment Goes Beyond Rivals in Myelofibrosis Anemia
You are invited to join hundreds of thousands of people touched by spina bifida, the most common congenital birth defect causing disability. There is no cure. This is a…
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October is Spina Bifida Awareness Month
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The 2022 Medscape Physician Suicide Report shows that physicians, compared to the general public, have double the rate of suicides. Amna Shabbir, a doctor of internal medicine, felt compelled…
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Are We Looking at a Physician Suicide Epidemic?
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Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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AI Can Predict Dementia Fifteen Years Prior to Symptoms Occurring
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Indie songwriter and singer Sufjan Stevens recently posted on, Tumblr, his favorite media platform, that he has been diagnosed with Guillain-Barré syndrome (GBS). Sufjan is now in the hospital…
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Sufjan Stevens Shares Guillain-Barré Syndrome Diagnosis Prior to New Album Release
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Tivdak, Seagen, and Genmab’s antibody-drug conjugate exceeded expectations. The partners said that a Data Monitoring Committee, independent of either company, noted that Tivdak’s overall survival rating met and even…
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Phase III Trial Results Show Significant Benefits in Patients with Metastatic Cervical Cancer
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Over 50% of non-small cell lung cancer patients (NSCLC) will experience a recurrence after surgery. The cancer recurs even after adjuvant chemotherapy after major surgery. ALINA’s data show that Alecensa…
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ICYMI: Alecensa Exhibited Unprecedented Results for Patients with Early-Stage Lung Cancer
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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Strange Symptoms Lead to Diagnosis of Cushing’s Disease
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Currently, almost half the patients with non-small cell lung cancer (NSCLC) will see their disease return despite aggressive chemotherapy treatment (adjuvant surgery). Adjuvant therapy is treatment that is given…
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Non-small Cell Lung Cancer: Alecensa Achieved Disease-Free Survival in Phase III Interim Analysis
A few words of comfort from NINDS, the National Institute of Neurological Disorders and Stroke as featured in MSN. This article is designed to help you cope with the pain,…
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A Helpful Guide to Mitigate Symptoms After a Migraine
Dozens of Ryan Wilson Palmer’s family and friends recently celebrated an early birthday after he received a devastating diagnosis of Creutzfeldt-Jakob disease. A few months ago 49-year-old Ryan Palmer of…
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Ryan’s Life Changed from being a Fire Fighter to Being Diagnosed with Creutzfeldt-Jakob Disease
Antibody-drug conjugates (ADC) are one of the fastest-growing anticancer drugs. BioNTech’s licensed ADC is now in Phase III. According to a report in BioSpace, BioNTech is attempting to compete with…
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BioNTech and DualityBio Moving ADC into Phase III for HER2 Low Breast Cancer
Beam Therapeutics (Beam) has developed a technique called base editing. The treatment is called BEAM 201. The first patient has been dosed according to a recent statement by the company…
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Beam Therapeutics Announces a First in Human Testing of Gene Editing Medicine for Cancer